臨床研究等提出・公開システム

Japanese

Date of registration	Mar. 20, 2019
Last modified on	Sept. 27, 2022
Trial ID	jRCTs031180398

Scientific Title	Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial (CGD-RIST2)
Public Title	A phase II study of RIC-SCT for CGD (CGD-RIST2)

Contact for Scientific Queries	Name	Sakaguchi Hirotoshi
	Affiliation	National Center for Child Health and Development
	Address	2-10-1, Okura, Setagaya-ku
	Telephone	+81-3-3416-0181
	E-mail	sakaguchi-hi@ncchd.go.jp
Contact for Public Queries	Name	Sakaguchi Hirotoshi
	Affiliation	National Center for Child Health and Development
	Address	2-10-1, Okura, Setagaya-ku
	Telephone	+81-3-3416-0181
	E-mail	sakaguchi-hi@ncchd.go.jp

Recruitment status	Recruiting

Anticipated date of first enrollment		Dec. 30, 2017
Actual date of first enrollment		May. 30, 2018
Target sample size		22
Study Type		Interventional
Study Design	allocation	single arm study
	masking	open(masking not used)
	control	no treatment control/standard of care control
	assignment	single assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	1) Diagnosed as chronic granulomatous disease 2) Planned to have stem cell transplantation 3) Donor for transplantation is one of the follows; a. HLA 5/6 (A, B, DR) or more matched related donor b. HLA 5/6 (A, B, DR) or more matched unrelated donor 4) Age at transplantation <25 years 5) ECOG Performance status: 0-2 6) Sufficient organ function, as follows; a. T-Bil <= 1.5 mg/dl b. Serum creatinine <= 0.8 mg/dl (age <5y), <= 1.2 mg/dl (age 5-9y), <= 1.5 mg/dl (10y or older) c. Ejection fraction 45% or better, and QTfc <0.45 sec 7) Obtained informed consent from a guardian of the patient
	Exclusion Criteria	1. Intracranial hemorrhage 2. Severe psychological disorders 3. Pregnant or suspected pregnancy 4. Receiving interferon gamma therapy within 4 weeks of the start of conditioning therapy 5. Not eligible for this study at the discretion of the investigator.
	Age Minimum	No limit
	Age Maximum	25age old not
	Gender	Both
Health Condition(s) or Problem(s) Studied		Chronic granulomatous disease
Intervention(s)		Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0
Health Condition(s) Keyword		Primary immunodeficiency
Intervention(s) Keyword
Health Condition(s) Code		D006105
Intervention(s) Code
Primary Outcome(s)		Event (graft failure/death) at day 100 after transplantation
Secondary Outcome(s)		1. Engraftment probability at day 30 2. Mortality at day 100 3. Incidence of infectious disease at week 8 4. Reconstruction of immune status 5. Time from transplantation to onset of acute GVHD 6. Reconstruction of neutrophil function

Primary Sponsor

Primary Sponsor

Primary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support / Secondary Sponsor
Secondary Sponsor

Source of Monetary Support	National Center for Child Health and Development
Secondary Sponsor	Not applicable

Name of Certified Review Board	Certified Review Board of National Center for Child Health and Development
Address	2-10-1, Okura, Setagaya-ku, Tokyo 157-8535 Japan, Tokyo
Telephone	+81-3-3416-0181
E-Mail	rinken@ncchd.go.jp
Approval Status	Approval
Date of approval	July. 26, 2018

Secondary ID(s)	UMIN000030647
Issuing Authority	University Hospital Medical Information Network Center

Countries of Recruitment（Except Japan）	None (Japan only)

History of Changes

No	Publication date
12	Sept. 27, 2022	(this page)	Changes
11	Sept. 15, 2022	Detail	Changes
10	April. 26, 2022	Detail	Changes
9	Feb. 22, 2022	Detail	Changes
8	May. 31, 2021	Detail	Changes
7	Mar. 22, 2021	Detail	Changes
6	Dec. 24, 2020	Detail	Changes
5	Dec. 16, 2020	Detail	Changes
4	May. 28, 2020	Detail	Changes
3	May. 26, 2020	Detail	Changes
2	Dec. 10, 2019	Detail	Changes
1	Mar. 20, 2019	Detail

List of change history